With experimental drugs, "right to try" can go wrong

Photo by Maria Kaloudi

Access to experimental drugs by terminally ill patients is a highly contested topic. Since 2014, several state legislatures, including in Texas, have passed "right to try" laws, which grant patient access to experimental drugs.

However, the laws remove patient protection mandated by the U.S. Food and Drug Administration. Without the FDA, patients may not accurately consider or have access to the data on the risk of taking the drug, may be charged an unnecessary amount for the drug or intervention and have no one to safeguard informed decisions.

"Right to try" laws theoretically increase the pool of experimental drugs available to patients, but they do very little to actually increase access and improve care. The laws were initially proposed in 2012 by the Goldwater Institute, a libertarian think tank in Arizona and subsequently passed by 28 states.

The laws give terminally ill patients access to drugs that have passed phase 1 of clinical trials without approval from the FDA or an ethics internal review board. But while phase 1 trials test the dosage of a drug, they do not always look into the drug's effectiveness or any possible side effects. This is an important distinction compared with drugs that have passed phase 2 or 3 trials, where efficacy is tested.

Moreover, patients can already obtain early access to clinical-trial interventions and drugs through the FDA's Expanded Access program. More than 99 percent of Expanded Access applications are accepted by the FDA.

The application process for Expanded Access has been justifiably critiqued as being arduous and lengthy — it can take more than 100 hours to complete — but the FDA has recently taken steps in the right direction. Recent revisions to the application process decreased the quantity of documentation required and severely reduced the amount of time it estimates it takes to complete the forms — to approximately 45 minutes. Despite this, patient advocates claim that the FDA system is a roadblock to real access to experimental drugs.

Rather than using these "right to try" laws to access drugs, advocacy groups should work with the FDA to make the already established Expanded Access program more straightforward for patients and physicians.

Although the "right to try" laws were written with good intentions, they do little to protect the patient and provide true access to a large population of patients. The laws lack the same level of risk-benefits assessment and informed consent that the FDA's Expanded Access program has. Physicians and patients are often not adequately informed about the risks of an experimental drug. The FDA also determines whether a patient can be charged for access to the drug, while "right to try" allows the pharmaceutical companies to deem the appropriate cost.

Removing the FDA from the process leaves patients vulnerable to unknown side effects, including increased suffering and hastened death.

To make the Expanded Access program easier and more straightforward for patients and physicians, the FDA has taken an important and significant step this past month. Hopefully, this will incentivize physicians and patients to work with the FDA rather than working around it. And hopefully it will also promote further collaboration between patient advocate groups and the FDA to fix other barriers to access.

Michelle Rubin

Graduate student, Baylor College of Medicine

Kirstin R.W. Matthews

Baker Institute fellow